Eight million pages, the Space Shuttle, and other highlights from BMWS 2019
by Marc Puich | November 15, 2019
If you’re producing cell therapy products for 10,000 patients a year, how many release tests will be required? If making a simple drug product such as aspirin requires about as much complexity as manufacturing a bicycle, then what’s the vehicular equivalent for a cell therapy product? Given all the complexities of personalized therapeutics, how does the road ahead really look?
At Biomanufacturing World Summit this past week, leaders at the forefront of producing cell and gene therapies gathered to talk about what’s really required to produce deeply individualized therapeutics at scale. It was a truly thought-provoking meeting. Here are a few of the highlights.
bluebird bio: scaling up and scaling out when one patient means one batch
Derek Adams, Chief Technology and Manufacturing Officer for bluebird bio provided an insightful keynote covering the gamut of learnings from their march to commercialization. When you’re treating just one cell therapy patient, the related numbers in terms of documents, FTE hours, and release tests are already sizeable. Scale to 10,000 patients, and some of the related numbers hit the “million” mark -- eight million pages of documentation, for starters.
(Support in managing this challenge is at the core of Vineti’s mission -- digital automation of the patient-centric supply chain and patient journey is truly required to manage and produce cell therapies at scale.)
Allogene: CMC skills are critical
Alison Moore, Chief Technology Officer at Allogene provided a very detailed look at the contrast between traditional biopharmaceutical products and cell therapies. Having transitioned to Allogene just nine months ago from Amgen, she highlighted the incredible amount of ecosystem innovation still required to scale this industry, especially as they target allogeneic therapies. As some of my colleagues have noted in a whitepaper on advanced therapy data management, CMC takes on an entirely new prominence in cell therapy. This presentation did a great job of breaking down the need for adapting CMC skills to advanced therapies, and the importance of data to drive repeatability, scalability, and ultimately safety and efficacy. With leaders like Alison Moore now joining startups at earlier stages (like Tim Moore at PACT), a large focus will be on manufacturability and scalability earlier in the product development lifecycle, with the goal of preventing approval delays, or launch issues once products are approved.
Bayer: CGT changes the value chain
Wolfram Carius, Executive Vice President Pharmaceuticals Supply, discussed Bayer’s increasing investment in the allogeneic cell therapy market, and what it means for them to transform their business to support these unique products. In cell and gene therapies, the value chain is distinct from the supply chain. This presentation outlined the ways that the cell therapy value chain drives close collaborations across previously siloed organizations. The digital enablers of this market are also new and distinct in many ways from prior pharmaceutical products, requiring invention in many areas to scale out these products.
GSK: CGT and spacecraft
Joseph Tarnowski, Ph. D., Senior Vice President, R&D, Medicinal Science & Technology, Cell & Gene Therapy Platform, reviewed GSK’s long history in this emerging space, with a pre-CAR-T approval of an autologous ex vivo gene therapy (Strimvelis) for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016. They are continuing to innovate, not just on the cell therapy manufacturing side, but also on the viral vector supply chain. This talk highlighted the relative cost of development and complexity in cell therapy -- if making aspirin is comparable to a bicycle, then producing cell therapies looks a lot more like the Space Shuttle.
PACT: the importance of continuous learning
Tim Moore, now the President and Chief Technical Officer at PACT Pharma, successfully led Kite Pharma’s CMC team in the successful launch of Yescarta in 2017. He is now taking his talent and experience to an even more complex cell therapy modality at PACT. This presentation made one reality clear -- even with approved CAR-T cell therapies now on the market for more than two years, the continuous application of lessons learned will be required for the next era of cell therapy production.
GSK: looking ahead
Much of the conference was spent discussing complexity -- but the challenges didn’t dampen the mood or the focus on finding solutions. The title of this talk summed it up well: in cell therapy, the future is bright!
Marc Puich has extensive experience in pharmaceutical manufacturing systems and cell therapy workflows, and is Vice President of Business Development at Vineti. If you’d like to see how Vineti’s Personalized Therapy Management (PTM) platform can help you solve your advanced therapy supply chain and workflow challenges, please contact us to schedule a demo.